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The purpose of this study is to collect long-term safety data in patients with metachromatic leukodystrophy (MLD) who are receiving HGT-1110 and have participated in Study HGT-MLD-070 through Week 40.

Condition or disease

Intervention/treatment

Phase

Metachromatic Leukodystrophy (MLD)

Biological: Recombinant human arylsulfatase A

Phase 1Phase 2

Detailed Description:

Metachromatic leukodystrophy (MLD) is an inherited, autosomal recessive disorder of lipid metabolism characterized by deficient activity of the lysosomal enzyme, arylsulfatase A (ASA). MLD is a rare disease that occurs in most parts of the world. The estimated overall incidence of the disease in the western world is approximately 1 in 100,000 live births that varies by geographic location. There are no approved therapies for MLD.

This study is a multicenter open-label study designed to evaluate safety and efficacy outcomes of HGT-1110 administered intrathecally in children with MLD who have participated in the dose escalation study, HGT-MLD-070, through Week 40 and are receiving study drug every other week (EOW).

Treatment groups will be identical to those in HGT-MLD-070, ie, patients assigned to Cohort 1 in Study HGT-MLD-070 will continue to receive a dose of 10 mg, patients assigned to Cohort 2 in Study HGT-MLD-070 will continue to receive a dose of 30 mg, and patients assigned to Cohorts 3 and 4 in Study HGT-MLD-070 will continue to receive a dose of 100 mg. Patients in Cohort 4 are to exclusively receive drug product produced with Process B in Study HGT-MLD-070 and will continue receiving this drug product in this study. Patients enrolled in this study from Cohorts 1 to 3 in Study HGT-MLD-070 were transitioned to Process B after all necessary approvals were obtained.

In HGT-MLD-071, all patients in the 10 mg dose cohort who experienced disease progression, as determined by the Investigator, increased to the 30 mg dose after agreement by the Medical Monitor. Based on the interim analysis results from HGT-MLD-070 (Cohorts 1-3), the dose of HGT-1110 will be increased to 100 mg for all patients in HGT-MLD-071 after all necessary approvals were obtained.

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Ages Eligible for Study:

up to 13 Years (Child)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Patient has participated through Week 40 in Study HGT-MLD-070.

Patient must have no safety or medical issues that contraindicate participation.

The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.

The patient has any known or suspected hypersensitivity to agents used for sedation or is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions.

The patient is pregnant or breast feeding.

The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 within 6 months prior to study enrollment or at any time during the study.